Research programme: gene editing therapeutics - CRISPR Therapeutics

Drug Profile

Research programme: gene editing therapeutics - CRISPR Therapeutics

Alternative Names: CRISPR-Cas9 gene therapies

Latest Information Update: 19 Apr 2017

Price : $50

At a glance

  • Originator CRISPR Therapeutics
  • Developer CRISPR Therapeutics; Genethon; Vertex Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene modulators; Immunomodulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Cancer; Haemoglobinopathies; Mucopolysaccharidosis I
  • Research Cystic fibrosis; Duchenne muscular dystrophy; Unspecified

Most Recent Events

  • 13 Apr 2017 University of California, University of Vienna, and Charpentier appeals decision by PTAB, which dismissed interference proceedings against Broad Institute, MIT, and Harvard University for a patent related to CRISPR-Cas9 genome editing technology, in USA
  • 28 Mar 2017 The European Patent Office announces its intention to grant a patent for CRISPR-Cas9 gene-editing technology to University of California, University of Vienna, and Charpentier
  • 10 Mar 2017 Preclinical trials in Cancer and Mucopolysaccharidosis I in USA (unspecified route)
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